Division of Pediatric Blood & Marrow Transplantation - Research
"We dare to suggest we can transform the practice of medicine... because we've done it before... many times."
—John Wagner, MD, Founding Director of the new Institute of Cell, Gene, and Immunotherapy
Our team within the Division of Blood and Marrow Transplantation (BMT) is focused on the integral relationship between clinical care and research. With world-class expertise, a culture of cross-disciplinary collaboration, and a proven track record of successfully bringing therapies from the lab to patients, our physicians and researchers are leading the field in developing treatments and cures for childhood cancers and rare diseases. We continue to break new ground in a variety of areas. We are leaders in alternative stem cell therapies, umbilical cord blood transplantation and immune-based therapies. We also provide novel treatment options for patients with rare, life-threatening conditions and work tirelessly to reduce transplant side effects and improve quality of life for patients after transplant. Our research is focused in three areas:
- Finding treatments and cures for the incurable
- Making transplant more successful
- Improving the long-term health and outcomes for our patients
Researchers in the Transplant Biology and Therapy program collaborate closely with scientists from the University of Minnesota's Masonic Cancer Center, Stem Cell Institute and Medical School. Our groundbreaking research has led to the development of treatments for leukemia, lymphoma, inherited genetic diseases, immune disorders and hemoglobinopathies.
We continue to push the boundaries of science to increase the understanding of pediatric cancers and other diseases that can be treated with blood and marrow transplantation (BMT). Together, we challenge the status quo and help to change the practice of medicine.
Faculty Researcher Profiles
Epidermolysis Bullosa (EB) Center
The ALD and Leukodystrophy Center at the University of Minnesota has unsurpassed experience treating leukodystrophy patients. Our physicians lead the field in several areas of research and continue to explore innovative ways to improve treatment success.
Mucopolysaccharidosis (MPS) Center
Our MPS Center is internationally recognized for our expertise, innovative treatment approaches, and research. Patients are cared for by experts in the areas of audiology, blood and marrow transplantation, cardiology, ear, nose and throat, endocrinology, genetics, neurology, neuropsychology, neuroradiology, ophthalmology, orthopeadic surgery, and pulmonology. This multidisciplinary team approach ensures that the care we provide best meets the unique needs of each patient and family.
Immunobiology and transplantation are the main research interest in the Blazar Laboratory. Project areas include: Graft-versus-host disease, regulatory T cells, immune recovery and regeration post-transplant, graft-versus-leukemia and genetic manipulations and reprograming of immature and mature cells.
The Bosnakovski lab is focused on tissue regeneration and understanding the molecular and cellular mechanisms of FSHD and sarcomas. The current work includes exploring the molecular and cellular effects of DUX4 expression in skeletal muscle and studying antifibrotic drugs on an FSHD mouse model. The lab is also modeling the initiation of CIC-DUX4 pediatric sarcomas and exploring novel epigenetic-targeted therapies for Ewing sarcomas.
Congenital heart defects and muscular dystrophies are common hereditary diseases affecting children, but effective treatments for most of these disorders are scarce. In the Chan Laboratory, they are working on deciphering the molecular regulation of how these diseases develop and establishing stem cell-based therapies to combat them.
The Laboratory of Experimental Tissue Regeneration, led by Dr. Michael Kyba, focuses on factors regulating tissue degeneration and regeneration, with a view towards using cell therapy for regenerative therapy.
The Panoskaltsis-Mortari laboratory is centered on the field of regenerative medicine. We study tissue engineering from stem cells using two approaches:
- Decellularized whole organ scaffolds integrated with sophisticated bioreactors
- 3D Bioprinting using extrusion, suspension, and laser-assisted techniques
The Tolar Laboratory focuses on finding new ways of treating children with lethal diseases. Additional research areas include:
- Reducing the negative effects of stem cell transplantation
- Creation and use of induced pluripotent stem cells
- Gene therapy using gene addition
- Gene editing